The latest data from a Phase I/II study shows that pegtibatinase can yield a rapid, sustained reduction in homocysteine, the protein behind the underlying cause of the rare disease.

Almost 80% of novel agent submissions from the biggest companies were approved in recent years, compared with a 60% approval rate for the rest of the industry, according to a Pink Sheet analysis of FDA approval decisions since 2021.

Public Company Edition: With the XBI up more than 9% in April, a few follow-on offerings –$23m to support Gamida Cell’s small product launch and $500m to fund Vaxcyte’s large clinical trial program – have made it to market. Also, China’s CytoMed launched the sixth biopharma IPO in the US this year.

The latest drug development news and highlights from the Pink Sheet’s US FDA Performance Tracker.